JP2010530239A5 - - Google Patents

Info

Publication number

JP2010530239A5

JP2010530239A5 JP2010512808A JP2010512808A JP2010530239A5 JP 2010530239 A5 JP2010530239 A5 JP 2010530239A5 JP 2010512808 A JP2010512808 A JP 2010512808A JP 2010512808 A JP2010512808 A JP 2010512808A JP 2010530239 A5 JP2010530239 A5 JP 2010530239A5

Authority

JP

Japan

Prior art keywords

nucleic acid

acid molecule

short interfering

interfering nucleic

double

Prior art date

2008-06-18

Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)

Granted

Links

• Espacenet
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• 150000007523 nucleic acids Chemical class 0.000 claims 22
• 230000002452 interceptive effect Effects 0.000 claims 20
• 108020004707 nucleic acids Proteins 0.000 claims 20
• 102000039446 nucleic acids Human genes 0.000 claims 20
• 239000002773 nucleotide Substances 0.000 claims 8
• 125000003729 nucleotide group Chemical group 0.000 claims 8
• 239000013604 expression vector Substances 0.000 claims 5
• 230000000692 anti-sense effect Effects 0.000 claims 3
• 230000000295 complement effect Effects 0.000 claims 3
• 230000035897 transcription Effects 0.000 claims 3
• 238000013518 transcription Methods 0.000 claims 3
• 208000023105 Huntington disease Diseases 0.000 claims 2
• 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
• 108091034117 Oligonucleotide Proteins 0.000 claims 2
• 108090000623 proteins and genes Proteins 0.000 claims 2
• 230000005026 transcription initiation Effects 0.000 claims 2
• 239000013598 vector Substances 0.000 claims 2
• 101001030705 Homo sapiens Huntingtin Proteins 0.000 claims 1
• JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 1
• 230000002238 attenuated effect Effects 0.000 claims 1
• 230000002950 deficient Effects 0.000 claims 1
• 229960003722 doxycycline Drugs 0.000 claims 1
• XQTWDDCIUJNLTR-CVHRZJFOSA-N doxycycline monohydrate Chemical compound O.O=C1C2=C(O)C=CC=C2[C@H](C)[C@@H]2C1=C(O)[C@]1(O)C(=O)C(C(N)=O)=C(O)[C@@H](N(C)C)[C@@H]1[C@H]2O XQTWDDCIUJNLTR-CVHRZJFOSA-N 0.000 claims 1
• 239000003814 drug Substances 0.000 claims 1
• 102000054185 human HTT Human genes 0.000 claims 1
• 230000000977 initiatory effect Effects 0.000 claims 1
• 238000004519 manufacturing process Methods 0.000 claims 1
• 239000008194 pharmaceutical composition Substances 0.000 claims 1
• 230000002265 prevention Effects 0.000 claims 1
• 239000002213 purine nucleotide Substances 0.000 claims 1
• 150000003212 purines Chemical class 0.000 claims 1
• 150000003230 pyrimidines Chemical class 0.000 claims 1
• 230000001105 regulatory effect Effects 0.000 claims 1
• 230000010076 replication Effects 0.000 claims 1
• 238000011808 rodent model Methods 0.000 claims 1
• 230000005030 transcription termination Effects 0.000 claims 1